After a year of huge headlines for children’s health, we’ve finally arrived at our last roundup of Children’s Hospital of Philadelphia research news for 2017. The latest developments cap off a benchmark year of discovery, with new advances in treating childhood blindness, novel initiatives to study traumatic brain injury, and wonderful stories from a variety of media outlets that highlight the efforts and accomplishments of our Mitochondrial Medicine Frontier Program and The Raymond G. Perelman Center for Cellular and Molecular Therapeutics. We think this edition’s collection of news items herald exciting things to come in 2018!

It was a big year for children’s health: We celebrated the U.S. Food and Drug Administration’s approval of the world’s first chimeric antigen receptor (CAR)-T cell therapy this September, followed closely by approval of the very first gene therapy to treat inherited blindness this month — both of which have their roots at Children’s Hospital of Philadelphia and the University of Pennsylvania. But besides the big headline-making breakthroughs (brilliant as they are), we wanted to know what other stories captivated our readers in 2017.

More than a decade of studies led by researchers at Children’s Hospital of Philadelphia and the University of Pennsylvania culminated this week with the U.S. Food and Drug Administration’s approval of an innovative one-time gene therapy product indicated for the treatment of patients with a rare, inherited form of retinal blindness.

The health and well-being of the children we treat truly starts at home where they live and play, and with the parents or caregivers whom they return to every day. At Children’s Hospital of Philadelphia Research Institute, Brittany Schliep, a clinical research finance manager, is fighting for healthier and happier homes by helping to prevent the impact of domestic violence (DV) on children in her hometown of Gloucester Township, N.J.

The year 2017 might be coming to a close, but research continues to ramp up at Children’s Hospital of Philadelphia, with exciting developments in the fields of brain science, hemophilia, gene therapy, and more. In this week’s roundup of headlines, we take a look at remarkable reports from CHOP and Penn Medicine about the brain’s ability to reorganize itself after limb amputation, the first U.S. effort to observe the use of medical cannabis for children with autism, and exciting innovations to improve sickle cell disease treatment presented at the 59th American Society of Hematology Annual Meeting and Exposition. Read on to discover more about these brilliant breakthroughs.

Editor’s Note: As PolicyLab’s 10th anniversary as a Center of Emphasis within Children’s Hospital of Philadelphia Research Institute arrives in 2018, we invited Director David Rubin, MD, MSCE, and Deputy Director Meredith Matone, DrPH, MHS, to reflect on PolicyLab’s progress and future directions. Read on to get a glimpse of the four inventive research portfolios that will achieve the Center’s “research-to-action” goals.

Curiosity is one of the driving traits of a stellar researcher, and throughout her career, pioneering sleep researcher Carole Marcus, MBBCh, had an unwavering fascination with sleep medicine. Upon her unexpected recent death, we take a look back over her years of exceptional leadership and clinical research as director of the Sleep Center at Children’s Hospital of Philadelphia and the CHOP Clinical and Translational Research Center/Center for Human Phenomic Science. She also worked closely with colleagues at the University of Pennsylvania as associate director of the Institute for Translational Medicine and Therapeutics, and was an outstanding and generous mentor for many fellows and faculty, having earned the CHOP Faculty Mentor Award in 2015.

Breakthroughs in basic science build the foundation for clinical research and our treatment of children’s health. Many basic scientists, however, find themselves wanting to play a more active role in connecting their lab discoveries from the bench to the bedside. The Office of Postdoctoral Affairs at Children’s Hospital of Philadelphia held a Q&A panel, “How to Break Into Translational Research As a Basic Scientist,” in October as part of their week-long, biannual Translational Research Workshop.

This first of December, we’re recognizing World Aids Day by sharing the latest research from Children’s Hospital of Philadelphia investigators who partnered with the University of Pennsylvania, the University of Botswana, and the Botswana Ministry of Health through the Botswana-UPenn partnership, in order to address sub-Saharan Africa’s HIV/AIDS epidemic. Alongside their findings published last week, our news roundup also includes special congratulations to Diva De León-Crutchlow, MD, on a “sweet” new award from Congenital Hyperinsulinism International, and novel research findings from our investigators who study cardiology, genetics, and puberty.